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Serious Side Effect Sidelines Gene-Edited Sickle Cell Therapy from Graphite Bio

MedCity News

Graphite Bio voluntarily paused a Phase 1/2 test of its gene-edited therapy for sickle cell disease after the first patient in the study developed a serious blood complication. The setback will also delay plans to reach the clinic with another genetic medicine in its pipeline.

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FDA Nod in Duchenne Helps Wider Swath of Patients With the Rare Muscle Disease

MedCity News

Corticosteroids are a standard first-line treatment for Duchenne muscular dystrophy, but these drugs have many side effects. FDA approval of Italfarmaco’s Duvyzat is the first nonsteroidal drug to pass the agency’s regulatory bar for treating this rare disease.

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Novartis warns of two patients deaths after dosing with gene therapy Zolgensma

MedCity News

Liver complications are a known side effect risk of Zolgensma and other genetic medicines that are delivered by engineered viruses. The two deaths reported following treatment with Novartis’s Zolgensma mark the first fatal cases of acute liver failure associated with the gene therapy.

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MHRA approves first gene therapy for infusion into the brain

European Pharmaceutical Review

The product, produced by biopharma company PTC Therapeutics, is approved for patients 18 months and over. Upstaza is administered through a minimally invasive neurosurgical stereotactic procedure used to treat several neurological disorders in paediatric and adult patients. Upstaza clinical trials.

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Regulators and HTAs tune in to patient preference data

Clarivate

Obtaining the preferences of patients and their caregivers is not a new concept in healthcare. Increasingly, however, industry guidance documents utilize the term ‘patient preference’ in relation to new drug development and benefit-risk assessment. Health technology assessment (HTA) bodies also have some appetite for this information.

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Newron says pivotal treatment-resistant schizophrenia trial is a go

pharmaphorum

The Italian biopharma said it had decided to press ahead with the new study after assessing results from 100 patients with TRS in open-label studies who received the glutamate modulator drug as an add-on to their current therapy for more than six months. in the evenamide group.

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Liver injuries prompt clinical hold on Sanofi BTK drug

pharmaphorum

billion buyout of Principia Biopharma has run into trouble, after the FDA placed it on partial clinical hold while a safety signal is investigated. One of the main assets of Sanofi’s $3.7 The hold on dosing applies to subjects in the trials who have received the drug for less than 60 days.